Phase I Dose Escalation of Iodine-131–Metaiodobenzylguanidine With Myeloablative Chemotherapy and Autologous Stem-Cell Transplantation in Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Consortium Study

Matthay, Katherine K.; Tan, Jessica; Villablanca, Judith G.; Yanik, Gregory A.; Veatch, Janet; Franc, Benjamin L.; Twomey, Eilish; Horn, Biljana; Reynolds, C. Patrick; Groshen, Susan; Seeger, Robert C.; Maris, John M.

doi:10.1200/jco.2005.03.6400

Cited by 174 publications

(114 citation statements)

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“…Incorporation of high-dose 131 I-MIBG treatment into HDCT/auto-SCT might be an option although it also results in late effects. [10][11][12][13][14][15] Matthay et al 15 showed that incorporation of high-dose 131 I-MIBG treatment into HDCT/auto-SCT was feasible and effective in patients with refractory neuroblastoma. However, no studies to date have incorporated high-dose 131 I-MIBG treatment into tandem HDCT/ auto-SCT for treating newly diagnosed high-risk patients.…”

Section: Discussionmentioning

confidence: 99%

Tandem high-dose chemotherapy and autologous stem cell transplantation in patients with high-risk neuroblastoma: Results of SMC NB-2004 study

Sung

Son

Lee

et al. 2012

Bone Marrow Transplant

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, 50 consecutive patients with high-risk neuroblastoma were assigned to receive tandem HDCT (high-dose chemotherapy)/auto-SCT after nine cycles of induction chemotherapy. CEC (carboplatin þ etoposide þ cyclophosphamide) regimen and TM (thiotepa þ melphalan)-TBI regimen (or TM regimen for stage 3 patients) were the first and second HDCT regimens. Local radiotherapy, differentiation therapy with 13-cis-retinoid acid and immunotherapy with interleukin-2 were given after tandem HDCT/auto-SCT. Of the 50 patients, 49 underwent a first HDCT/auto-SCT and 47 underwent a second HDCT/auto-SCT. The tumor relapsed or progressed in 14 patients, secondary malignancy developed in one patient and one patient died from chronic lung disease. Therefore, 34 patients remained event free with a median follow-up of 54.5 months (range, 14-94 months) from diagnosis. The probabilities of 5-year OS and EFS for all 50 patients were 77.0% (95% confidence interval (CI), 63.7-90.3) and 71.4% (95% CI, 58.7-84.1), respectively. However, all patients who remained event free for 43 years after tandem HDCT/auto-SCT experienced late adverse effects. Chemotherapeutic dose-escalation strategy using tandem HDCT/auto-SCT was very encouraging for survival. However, further studies incorporating newer treatment modalities are needed to reduce late adverse effects without jeopardizing the survival rate.

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Section: Discussionmentioning

confidence: 99%

Tandem high-dose chemotherapy and autologous stem cell transplantation in patients with high-risk neuroblastoma: Results of SMC NB-2004 study

Sung

Son

Lee

et al. 2012

Bone Marrow Transplant

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“…The only dose-limiting toxicity (myelosuppression) of 131 I-MIBG treatment can be overcome through the use of SCT. Yanik et al [27] and Matthay et al [28] demonstrated that highdose 131 I-MIBG treatment could feasibly be incorporated into HDCT/autoSCT. High-dose 131 I-MIBG treatment has been recently used to reduce tumor burden prior to alloSCT [29,30].…”

Section: Discussionmentioning

confidence: 99%

Reduced‐intensity allogeneic stem cell transplantation for children with neuroblastoma who failed tandem autologous stem cell transplantation

Sung

Park

Chueh

et al. 2011

Pediatric Blood & Cancer

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INTRODUCTIONHigh-dose chemotherapy and autologous stem cell transplantation (HDCT/autoSCT) has improved the outcome of high-risk neuroblastoma (NB). However, the 3-year event-free survival rates in the randomized trials by the Children's Cancer Group and the German Society of Pediatric Oncology and Hematology were 34% and 47%, respectively, which is still unsatisfactory [1,2]. Recently, a few investigators have shown that tandem HDCT/autoSCT may be a feasible approach to further improve the outcome [3][4][5][6]. Unfortunately, however, about half of patients still die from treatment failure even after tandem HDCT/autoSCT. The major cause of treatment failure is tumor relapse. Conventional chemotherapy has been ineffective in these patients, and there has been no realistic chance for cure with conventional options alone [7][8][9].In this context, allogeneic SCT (alloSCT) is being investigated as a potential curative treatment option for patients who failed HDCT/autoSCT because it offers a graft-versus-tumor (GVT) effect not seen in HDCT/autoSCT. The graft-versus-leukemia (GVL) effect is a widely accepted major component of alloSCT in leukemia [10], and there is emerging evidence for a GVT effect in solid tumors [11]. A GVT effect has been also demonstrated in patients with advanced NB who received alloSCT [12][13][14][15]. However, regimen-related mortality following standard alloSCT with an intensive conditioning regimen may be extremely high in patients who have been already heavily treated [16][17][18].In recent years, several groups of investigators have developed reduced-intensity conditioning (RIC) regimens that lead to engraftment of donor lymphoid and hematopoietic stem cells without the extra-hematopoietic toxicities of standard myeloablative conditioning, while conserving the GVL or GVT effect. This reduced regimen-related toxicity may make reduced-intensity alloSCT (RI alloSCT) especially suitable for patients at high-risk of regimen-related mortality, particularly previous tandem HDCT/ autoSCT recipients. In adults, striking GVT effects after RI alloSCT have been described in refractory breast cancer and renal cell carcinoma [19,20]. However, reports concerning the possible GVT effect of RI alloSCT remain very limited in pediatric solid tumors, particularly in NB [21]. In this context, we evaluated the feasibility and efficacy of RI alloSCT in patients with NB who failed tandem HDCT/autoSCT. PATIENTS AND METHODS PatientsFrom March 2008 to March 2009, patients with NB who failed tandem HDCT/autoSCT were eligible. Patient with grade III/IV comorbid organ dysfunction prior to RI alloSCT was excluded from the study. This prospective study was approved by Samsung Medical Center Institutional Review Board, and informed consent was obtained from all parents/guardians. Treatment Prior to RI AlloSCTConventional chemotherapy was administered in order to reduce the tumor burden as much as possible prior to transplant. A salvage chemotherapy regimen was selected according to the regimen used prior to relapse, tol...

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“…The importance of dose escalation studies in answering these questions was emphasized (33,34). Also briefly discussed were the growing use of 90 Y-labeled microsphere and particle treatment for liver metastases and primary tumors and FDA-approved therapies for bone metastases, including 153 Sm-EDTMP (ethylenediaminetetramethylene phosphoric acid), 89 Sr, and, most recently, 223 Ra-dichloride.…”

Section: A Nuclear Medicine Physician Perspectivementioning

confidence: 99%

Targeted Radionuclide Therapy: Proceedings of a Joint Workshop Hosted by the National Cancer Institute and the Society of Nuclear Medicine and Molecular Imaging

et al. 2014

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The growing interest in targeted radionuclide therapy (TRT) for a broad range of applications is shared by a diverse group of medical professionals, including but not limited to physicians and basic scientists in several fields, as well as members of industry, regulatory bodies, and patients. However, no organizational structure is available to regularly bring these stakeholders together to discuss the latest findings and the most productive strategies to ensure that the potential benefits of TRT are realized.

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Phase I Dose Escalation of Iodine-131–Metaiodobenzylguanidine With Myeloablative Chemotherapy and Autologous Stem-Cell Transplantation in Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Consortium Study

Abstract: 131I-MIBG with myeloablative chemotherapy is feasible and effective for patients with neuroblastoma exhibiting de novo resistance to chemotherapy.

Cited by 174 publications

References 20 publications

Tandem high-dose chemotherapy and autologous stem cell transplantation in patients with high-risk neuroblastoma: Results of SMC NB-2004 study

Tandem high-dose chemotherapy and autologous stem cell transplantation in patients with high-risk neuroblastoma: Results of SMC NB-2004 study

Reduced‐intensity allogeneic stem cell transplantation for children with neuroblastoma who failed tandem autologous stem cell transplantation

Targeted Radionuclide Therapy: Proceedings of a Joint Workshop Hosted by the National Cancer Institute and the Society of Nuclear Medicine and Molecular Imaging

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